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Homology is based on groundbreaking science that harnesses the naturally occurring process of homologous recombination. This non-nuclease-based approach offers clear advantages in its precision, efficiency and on-target in vivo editing of genetic mutations. Homology obtained an exclusive worldwide license to this technology platform, which is based on the pioneering research of Saswati Chatterjee, Ph.D., Professor of Virology at the Beckman Research Institute at the City of Hope in California, member of the Recombinant DNA Advisory Committee (RAC) to the Office of the Director, National Institutes of Health (NIH) and former charter member of the Therapeutic Approaches to Genetic Diseases Study Section of the NIH. Dr. Chatterjee and her team led the first adeno-associated virus (AAV) vector-mediated gene transfer studies into human hematopoietic stem cells and subsequently identified and isolated a series of naturally-occurring AAVs from human CD34+ cells.
Akaza Research is a Waltham, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Korro Bio is an RNA editing company focused on the discovery and development of a new class of precision genetic medicines for both rare and common diseases. The company`s proprietary and modular platform, OPERA™ (Oligonucleotide promoted editing of RNA), builds on a deep understanding of ADAR biology, and combines data-driven design, oligo discovery and chemistry with clinically validated delivery vehicles, to achieve highly selective RNA editing. This unique technology enables the development of RNA editing therapeutics that deliver the functional benefits of gene therapy with a reversible, transient, titratable and specific treatment regimen, offering the potential to propel genetic medicine beyond rare genetic diseases into larger patient populations with common diseases. Korro is based in Cambridge, Mass.
GeneSage is a San Francisco, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Shape Therapeutics (ShapeTx) is a development-stage biotechnology company establishing itself as the leader in next-generation gene therapy.